iPSC-based disease modeling and drug discovery in cardinal neurodegenerative disorders

Hideyuki Okano; Satoru Morimoto

doi:10.1016/j.stem.2022.01.007

iPSC-based disease modeling and drug discovery in cardinal neurodegenerative disorders

Cell Stem Cell. 2022 Feb 3;29(2):189-208. doi: 10.1016/j.stem.2022.01.007.

Authors

Hideyuki Okano¹, Satoru Morimoto²

Affiliations

¹ Department of Physiology, Keio University School of Medicine, 35 Shinanomachi, Shinjuku-ku, Tokyo, Japan; Laboratory for Marmoset Neural Architecture, RIKEN Center for Brain Science, Wako-shi, Saitama 351-0198, Japan. Electronic address: hidokano@keio.jp.
² Department of Physiology, Keio University School of Medicine, 35 Shinanomachi, Shinjuku-ku, Tokyo, Japan.

PMID: 35120619
DOI: 10.1016/j.stem.2022.01.007

Abstract

It has been 15 years since the birth of human induced pluripotent stem cell (iPSC) technology in 2007, and the scope of its application has been expanding. In addition to the development of cell therapies using iPSC-derived cells, pathological analyses using disease-specific iPSCs and clinical trials to confirm the safety and efficacy of drugs developed using iPSCs are progressing. With the innovation of related technologies, iPSC applications are about to enter a new stage. This review outlines advances in iPSC modeling and therapeutic development for cardinal neurodegenerative diseases, such as amyotrophic lateral sclerosis, Parkinson's disease, and Alzheimer's disease.

Keywords: AD; ALS; Alzheimer’s disease; PD; Parkinson’s disease; amyotrophic lateral sclerosis; disease modeling; drug repositioning; iPSCs; induced pluripotent stem cells; stratification.

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

Amyotrophic Lateral Sclerosis* / drug therapy
Cell- and Tissue-Based Therapy
Drug Discovery
Humans
Induced Pluripotent Stem Cells* / pathology
Neurodegenerative Diseases* / drug therapy
Neurodegenerative Diseases* / pathology